Gene editing in cancer treatment.

Healthy vs Leukemia blood. (source: SCIGOGO)

Everything is a fad, until it changes an industry.

TV was a fad, but then it killed the radio star.

The internet was a toy for nerds, now it runs the world.

Even the car was originally a gimmick, but I’ve never jumped into my horse drawn cart and clip clopped my way through a macca’s drive through.

The only constant is change, with huge payoff for those getting in early on an industry changing technology. But people are wary of change. People want to be comfortable. People don’t want risk.

An English team of doctors and researchers took the risk to save two young infants. In doing so they cured the infants cancer when nothing else would. The result could change cancer treatment, while the payoff for getting in early isn’t measured in money, but lives saved.

The cancer

The two infants aged 11 and 16 months had a severe form of leukaemia, termed acute lymphocytic leukaemia (ALL). ALL is the most common cancer in children, in this case effecting the B-cells of the immune system.

CAR-T cells

The infants lives were saved with a novel method that had never been used in humans before. Doctors edited donor T-cells (cells of the immune system) to become CAR-T cells. This allows the cells to recognise and latch onto specific proteins that are only on the surface of B-cells, healthy or cancerous.

The resulting cells, termed CART19 cells, can seek out and destroy the cancerous cells. These modified cells had been trialled with success in the past.

What’s the difference here? The modified cells are from a donor and not the host. This means that the donor T-cells will recognise the hosts body as non-self and attack it. In addition, the host immune system and anti-leukaemia drugs will attack the donor CART19 cells in return.


TALEN proteins cutting out a gene. (source: Labomic)

To overcome this, Qasim and colleagues created the universal CART19 cell by editing out two genes that allow the drug to recognise and attack the host body as non-self and be attacked in return. To do this TALEN proteins were engineered to bind to the exact positions of these genes and then cut them out of the T-cells genome. BOOM! The UCART19 cell was born and boy did it work.

Within 28 days both patients were in remission and have been for over a year.

Moving forward

This is special, because it becomes a so called “off the shelf” alternative. It helps not only immunosuppressed cancer sufferers, but also drastically reduces the cost involved in creating personalised therapies, which were upwards of $50,000 a dose, but now are as little as $4000.

The very idea of gene editing human cells strikes fear into many, but with such striking results the only question is why not embrace the change. Will the industry be slow moving? Or will it react quickly? The payoff for adopting this new method years before it may have otherwise become mainstream might well be measured in lives saved.

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